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Myelodysplastic SyndromePhase 2NCT07463820

A Trial Comparing Three Different Treatment Options for Adults With Low-Risk Myelodysplasia and Anemia (A MyeloMATCH Treatment Trial)

A Phase 2 study testing Biospecimen Collection, Bone Marrow Aspiration and Emavusertib in people with Myelodysplastic Syndrome, run by National Cancer Institute (NCI).

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Official record
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Trial summary

This phase II MyeloMATCH treatment trial tests luspatercep with or without epoetin alfa or emavusertib for the treatment of low risk myelodysplasia and anemia. Biological therapies, such as luspatercep, use substances made from living organisms that may attack specific cancer cells and stop them from growing or kill… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myelodysplastic Syndrome
What is being tested?
Biospecimen Collection, Bone Marrow Aspiration and Emavusertib
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
270 participants
Where
No sites listed yet
Study dates
18 Dec 2026to 31 Dec 2027
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Participants must have been registered to Master Screening and Re-Assessment Protocol (MYELOMATCH) prior to consenting to this study. Participants must have been assigned to this clinical trial, via MATCHBox Protocol Assignment Team, prior to registration to this study. Participants must have agreed to have specimens submitted for translational medicine and must be offered the opportunity to submit biosamples for banking for future research as per MYELOMATCH
  • Participants must have pathologically confirmed MDS according to World Health Organization (WHO) 2022 classification, without excess blasts
  • Lower risk MDS with a revised international prognostic scoring system (IPSS-R) score of less than or equal to 3.5
  • Symptomatic anemia defined as either red blood cell (RBC) transfusion dependent, or hemoglobin < 95 g/L with subjective fatigue or dyspnea
  • Age 18 years or greater
  • No prior therapy for myelodysplasia; including no luspatercept, imetelstat, emavusertib, lenalidomide, hypomethylating or immunosuppressive agents. Patients may have received up to 2 prior doses of erythropoietin stimulating agents (epoetin alfa or darbepoetin) if at least 8 weeks have elapsed between the prior therapy and study enrollment
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  • No concurrent radiotherapy, biological, or any other investigational agents within 30 days prior to enrollment
  • Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2
  • Absolute neutrophil count ≥ 0.5 x 10^9/L
  • Platelet count ≥ 50 x 10^9/L
  • Bilirubin ≤ 3.0 x upper limit of normal (ULN)
  • If confirmed Gilbert's, eligible providing direct bilirubin ≤ 3.0 x ULN
  • Alanine aminotransferase (ALT) ≤ 3.0 x ULN
  • Erythropoeitin ≤ 500 IU/L
  • Creatinine clearance > 30 mL/min
  • Creatinine clearance to be measured directly by 24-hour urine sampling or as calculated by Cockcroft and Gault equation
  • Participant consent must be appropriately obtained in accordance with applicable local and regulatory requirements. Each participant must sign a consent form prior to enrollment in the trial to document their willingness to participate. A similar process must be followed for sites outside of Canada as per their respective cooperative group's procedure
  • Participants must be accessible for treatment and follow-up. Participants enrolled on this trial must be treated and followed at the participating center. Investigators must assure themselves the participants enrolled on this trial will be available for complete documentation of the treatment, adverse events, and follow-up. Participants must agree to return to their primary care facility for any adverse events which may occur through the course of the trial
  • In accordance with Canadian Cancer Trials Group (CCTG) policy, protocol treatment is to begin within 7 working days of participant enrollment
  • Males and females of reproductive potential must have agreed to use a highly effective contraceptive method during study treatment and for 3 months afterwards. A participant is considered to be of "childbearing potential" if they have had menses at any time in the preceding 12 consecutive months. In addition to routine contraceptive methods, "effective contraception" also includes celibacy and surgery intended to prevent pregnancy (or with a side-effect of pregnancy prevention) defined as a hysterectomy, bilateral oophorectomy or bilateral tubal ligation, or vasectomy/vasectomized partner. However, if at any point a previously celibate participant chooses to become sexually active during the time period for use of contraceptive measures, they are responsible for beginning contraceptive measures. Participants of childbearing potential will have a pregnancy test to determine eligibility as part of the Pre-Study Evaluation; this may include an ultrasound to rule-out pregnancy if a false-positive is suspected. For example, when beta-human chorionic gonadotropin is high and partner is vasectomized, it may be associated with tumor production of human chorionic gonadotropin (hCG), as seen with some cancers. Participant will be considered eligible if an ultrasound is negative for pregnancy

Who cannot join

  • Participants with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial
  • MDS with del(5q)
  • Concurrent use of hematopoietic growth factors (other than protocol specified)
  • Known hypersensitivity to either luspatercept, erythropoietin, or emavusertib therapy
  • Participants who are unable to take oral medication regularly, with active gastroparesis, short gut syndrome, or other malabsorption syndrome
  • Active, uncontrolled bacterial, fungal, or viral infection within 14 days prior to enrollment.
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  • Participants with hepatitis B core antibody positive suggestive of past infection are eligible if they are hepatitis B virus (HBV) deoxyribonucleic acid (DNA) negative and concurrently treated with anti-viral therapy. Participants who are hepatitis B surface antigen positive should be referred for appropriate care and considered eligible if hepatitis B DNA becomes negative.
  • Participants with a history of hepatitis C which has been treated and is no longer active are eligible. Patients who have not been treated should be referred for appropriate care.
  • HIV-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial.
  • Participants on prophylactic antimicrobials need not be excluded, although specific agents may be excluded for drug-to-drug interactions or overlapping toxicities.
  • Participants who do not meet these eligibility criteria should be referred for appropriate care and may be considered in the future if eligibility criteria are met
  • Participants with serious illnesses or medical conditions which would not permit the participant to be managed according to protocol including but not limited to:
  • Uncontrolled hypertension, defined as repeated systolic blood pressure greater than 160 and/or diastolic greater than 100 mmHg despite adequate treatments,
  • Patients with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification. To be eligible for this trial, patients should be class II or better
  • Iron deficiency defined as ferritin of < 50 ug/L
  • Live attenuated vaccination administered within 30 days prior to enrollment
  • Note: Seasonal vaccines for influenza are generally inactivated vaccines and are allowed. Intranasal vaccines are live vaccines and not allowed
  • Participants who are pregnant or breast-feeding are excluded from this study because luspatercept was found to be hazardous to the fetus in pregnant animals and was found in the milk of treated lactating rats. Emavusertib and epoetin alfa have not been evaluated in pregnant or lactating individuals, and potential risks to the fetus are unknown

About this study

Official title: A Phase II Study of Combination Therapy With Luspatercept in Lower Risk Myelodysplasia: A Tier 1 MyeloMATCH Substudy

This phase II MyeloMATCH treatment trial tests luspatercep with or without epoetin alfa or emavusertib for the treatment of low risk myelodysplasia and anemia. Biological therapies, such as luspatercep, use substances made from living organisms that may attack specific cancer cells and stop them from growing or kill them. Epoetin alfa is a substance that is made in the laboratory and stimulates the bone marrow to make red blood cells. Emavusertib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving luspatercep with or without epoetin alfa or emavusertib may be effective for treating patients with low risk myelodysplasia and anemia.

Read the detailed description

PRIMARY OBJECTIVE:

  1. To assess the erythroid response rate at 24 weeks (Hematologic Improvement - Erythroid) using revised international working group 2018 criteria (Platzbecker 2019b), in patients with myelodysplastic syndrome (MDS) with previously untreated lower risk disease and symptomatic anemia, when treated with luspatercept based combination or mono-therapy, according to the presence or absence of specific spliceosome mutations.

SECONDARY OBJECTIVES:

  1. Transfusion-free survival. II. Duration of the erythroid response. III. Depth of the erythroid response. IV. Bi- or tri-lineage responses. V. Safety and tolerability. VI. Overall and leukemia-free survival. VII. Minimal residual disease (MRD) with next generation sequencing and flow cytometry VIII. Comparison of Hematologic Improvement - Erythroid and safety between arms 2 and 3 (if both arms are positive).

IX. Comparison of Hematologic Improvement - Erythroid and safety between ring sideroblast + and - populations.

OUTLINE: This is a dose-escalation study of luspatercept in combination, dependent upon arms assignments, with fixed dose epoetin alfa or emavusertib. Patients are randomized to 1 of 3 arms.

ARM 1: Patients receive luspatercept subcutaneously (SC) on day 1 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients undergo bone marrow aspiration and blood sample collection throughout the study.

ARM 2: Patients receive luspatercept SC on day 1 and epoetin alfa SC once weekly (QW) for each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients undergo bone marrow aspiration and blood sample collection throughout the study.

ARM 3: Patients receive luspatercept SC on day 1 and emavusertib orally (PO) twice daily (BID) on days 1-21 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients undergo bone marrow aspiration and blood sample collection throughout the study.

After completion of study treatment, patients are followed up at 4 weeks and every 3 months until loss of response and/or the development of transfusion dependence then every 6 months thereafter or every 6 months for patients not in HI-E and are transfusion dependent or who have progressed/relapsed.

Being tested
Biospecimen Collection, Bone Marrow Aspiration, Emavusertib, Epoetin Alfa, Luspatercept
Medical subject headings
Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Biospecimen CollectionProcedure or surgery

    Also called Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection

    Undergo blood sample collection

  • Bone Marrow AspirationProcedure or surgery

    Undergo bone marrow aspiration

  • EmavusertibBiological

    Also called AU 4948, AU-4948, CA 4948, CA-4948

    Given PO

  • Epoetin AlfaBiological

    Also called EPO, Epoetin alfa-epbx, Epogen, Eprex

    Given SC

  • LuspaterceptBiological

    Also called ACE 536, ACE-536, ACE536, Luspatercept-aamt

    Given SC

Study groups in NCT07463820
Study groupRoleWhat they receive
Arm 1 (Luspatercept)ExperimentalPatients receive luspatercept SC on day 1 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients undergo bone marrow aspiration and blood sample collection throughout the study.
Receives: Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration; Biological: Luspatercept
Arm 2 (Luspatercept with epoetin alfa)ExperimentalPatients receive luspatercept SC on day 1 and epoetin alfa SC QW for each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients undergo bone marrow aspiration and blood sample collection throughout the study.
Receives: Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration; Biological: Epoetin Alfa; Biological: Luspatercept
Arm 3 (Luspatercept and emavusertib)ExperimentalPatients receive luspatercept SC on day 1 and emavusertib PO BID on days 1-21 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients undergo bone marrow aspiration and blood sample collection throughout the study.
Receives: Procedure: Bone Marrow Aspiration; Biological: Emavusertib; Biological: Luspatercept

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Erythroid response rate

    Defined by the total number of patients with hematologic improvement-erythroid (HI-E) response divided by the total number of randomized patients in each arm. The comparison of the HI-E rate will be done using a Cochran-Mantel-Haenszel test stratified for the stratification factors at randomization based on the intent-to-treat population. The difference in the response rates and the corresponding one-sided 90% confidence limit will be calculated.

    Measured: At 24 weeks

Secondary outcomes (7)

  1. Transfusion free survival

    Defined as from achieving transfusion independence to the first day of transfusions after becoming transfusion dependent as defined by International Working Group (IWG) 2018 criteria for those who are transfusion dependent at enrollment or time to first day of transfusions as defined by IWG 2018 criteria for those nor transfusion dependent at enrollment.

    Measured: Up to 2 years

  2. Duration of erythroid response

    Measured: From meeting criteria for HI-E response to progression or relapse as defined by IWG 2018 criteria, up to 2 years

  3. Tri-lineage response

    Tri-lineage response involves erythroid, neutrophil, and platelet response per IWG 2018 criteria.

    Measured: Up to 2 years

  4. Bi-lineage response

    Any two of the responses for erythroid, neutrophil, and platelet response per IWG 2018 criteria.

    Measured: Up to 2 years

  5. Incidence of adverse events

    Measured: From the time of first treatment, up to 2 years

  6. Overall survival

    Measured: From enrollment to death from any cause, up to 2 years

  7. Overall leukemia free survival

    Measured: From enrollment to initial diagnosis of acute leukemia or death from any cause, up to 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Mar 2026
  2. Study start18 Dec 2026 (estimated)
  3. Primary completion31 Dec 2027 (estimated)
  4. Study completion31 Dec 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
270 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07463820
Sponsor's protocol ID
NCI-2026-01399
Other IDs
NCI-2026-01399, MM1MDS-CTG02, MM1MDS-CTG02, U10CA180863
Status last verified
Sep 2026
First submitted
10 Mar 2026
Last updated
22 Sep 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Biospecimen Collection, Bone Marrow Aspiration and Emavusertib in people with Myelodysplastic Syndrome, run by National Cancer Institute (NCI).

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 20 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 22 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Erythroid response rate (time frame: At 24 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07463820.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Sep 2026.

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